Bleeding recurrence in patients with gastrointestinal vascular malformation after thalidomide

Abstract

Thalidomide may be used for the treatment of gastrointestinal vascular malformation (GIVM), but the long-term response and adverse effects are unknown. Aim to study the recurrence rate of GIVM bleeding after thalidomide treatment, the response to treatment, and the adverse effects.This was a retrospective study of 80 patients with GIVM treated with thalidomide between November 2003 and November 2013. Patients received a course of 100 mg/day of thalidomide for 4 months and were followed up for at least 1 year. The response rate during follow-up, the recurrence rate after the 1st course of treatment, and the rate of retreatment were assessed. Comorbidities, the need for blood transfusion, yearly bleeding episodes, hemoglobin levels, hospitalization after thalidomide treatment, and the rate of adverse effects were also examined.The overall response rate during follow-up was 79.5% (62/78). The recurrence rate was 21.0% after the 1st course of thalidomide. The response rate of retreatment was 100%. After thalidomide treatment, yearly blood transfusion amounts, yearly bleeding episodes, and yearly hospitalization numbers were significantly decreased, while hemoglobin levels were significantly increased (P < 0.001). Adverse effects were observed in 60.0% (48/80) of the patients. Serious adverse effects were reported in 31.3% (25/80). The overall response rate was 76.7% (23/30) in 30 patients with comorbidities, while the rate was 78.0% (39/50) in patients without comorbidities (P = 0.55). The rate of serious adverse effects was similar between the comorbidities (33.3%) and no-comorbidities groups (30.0%) (P = 0.76).Thalidomide showed a good response rate and low adverse effect rate in patients with recurrent gastrointestinal bleeding due to GIVM.

Thalidomide for refractory gastrointestinal bleeding from vascular malformations in patients with significant comorbidities

“…Whereas the only randomized controlled trial so far by Ge et al[ 10 ] from China found a response rate of 71.4%, patients with cardiac, pulmonary, hepatic, renal, and other comorbidities were excluded from the study. In a follow-up retrospective study by the same group with 80 patients, the response rate to thalidomide in patients with or without comorbidities was 76.7% and 78% respectively, although this study again excluded patients with severe comorbidities such as cirrhosis and severe cardiac and renal conditions[ 14 ]. Therefore, it is unclear whether these results could be applied to other patient populations with significant comorbidities.…”

Novel Use of Low-dose Thalidomide in Refractory Gastrointestinal Bleeding in Left Ventricular Assist Device Patients

“…In studies involving non-LVAD patient populations, recurrent GIB following discontinuation of thalidomide therapy is well-documented and affects up to 20% of patients (16, 17). However, this has not been previously examined in LVAD patients.…”

Diagnosis and therapeutic strategies for small bowel vascular lesions

“…They found a significant reduction in bleeding episodes in the thalidomide group, compared with the control group after a mean follow-up period of 39 months. Similarly, Chen et al[62] reported that a significant reduction in bleeding episodes was confirmed in approximately 80% of the patients who received a course of 100 mg/d of thalidomide for 4 mo during a follow-up period of at least one year. However, previous clinical trials reported high rates of adverse events including fatigue, constipation, peripheral neuropathy, leukopenia and liver toxicity[62,63].…”

“…Similarly, Chen et al[62] reported that a significant reduction in bleeding episodes was confirmed in approximately 80% of the patients who received a course of 100 mg/d of thalidomide for 4 mo during a follow-up period of at least one year. However, previous clinical trials reported high rates of adverse events including fatigue, constipation, peripheral neuropathy, leukopenia and liver toxicity[62,63]. Together with the risk of birth defects associated with the use of thalidomide, these findings suggest that the clinical usefulness of thalidomide is likely to be limited to a small number of patients.…”